On 21 March 2012, orphan designation (EU/3/12/973) was granted by the European Commission to NDA Regulatory Science Ltd, United Kingdom, for recombinant human beta-glucuronidase for the treatment of mucopolysaccharidosis type VII (Sly syndrome).
The sponsorship was transferred to NDA Group AB, Sweden, in December 2013 and subsequently to Ultragenyx UK Limited, United Kingdom, in May 2015.
Recombinant human beta-glucuronidase for the treatment of mucopolysaccharidosis type VII (Sly syndrome) has been authorised in the EU as Mepsevii since 23 August 2018.
This medicine is now known as vestronidase alfa.
Recombinant human beta-glucuronidase (vestronidase alfa)
|Disease / condition||
Treatment of mucopolysaccharidosis type VII (Sly syndrome)
|Date of decision||
|Orphan decision number||
Review of designation
Sponsor's contact details
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
- Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe;
- European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.